Master Framework and Pragmatic Clinical Trial for Relapse or Refractory Acute Myeloid Leukemia

Project Information

IMPACT- AML

Grant agreement ID: 101104421

DOI

10.3030/101104421

EC signature date 27 April 2023

Start date 1 April 2023

End date 31 March 2028

Funded under

• Health

Total cost

€ 5 988 402,50

EU contribution

€ 5 988 402,50

Coordinated by

ISTITUTO ROMAGNOLO PER LO STUDIO DEI TUMORI DINO AMADORI - IRST SRL
Italy

Periodic Reporting for period 1 - IMPACT- AML (Master Framework and Pragmatic Clinical Trial for Relapse or Refractory Acute Myeloid Leukemia)

Reporting period: 2023-04-01 to 2024-09-30

Summary of the context and overall objectives of the project

Acute Myeloid Leukemia (AML) is an aggressive blood cancer with a poor prognosis, especially for patients with relapse or treatment resistance (R/R AML). Current treatment options are limited, highlighting the urgent need for better therapies and broader access to quality care. The IMPACT-AML project addresses these challenges with an inclusive, patient-centered approach to improve outcomes for R/R AML patients in Europe and beyond.

IMPACT-AML's primary goal is to build a collaborative framework where all R/R AML patients can be enrolled in registries or trials, regardless of background. This framework is supported by the STREAM platform, which standardizes and analyzes clinical data, facilitating Randomized Pragmatic Clinical Trials (RPCT) to improve treatment strategies and guide future care.

The project also focuses on biobanking, developing SOPs for tissue storage and MRD analysis to support translational research. A key aspect of IMPACT-AML is the RPCT, comparing low- and high-intensity rescue therapies, involving 340 patients across Europe for a representative dataset.

IMPACT-AML addresses vulnerable groups, including pediatric patients and the elderly, ensuring they are not overlooked. Patient and caregiver involvement is central, with partners like ALAN actively engaging to enhance patient experiences.

The project also aims to make AML care accessible across different healthcare systems, including lower-resource settings. Telemedicine pilots in countries like Ukraine and Tanzania extend AML care beyond Europe, promoting collaboration and reducing health disparities.

Through a network of clinical partners, biotech companies, patient organizations, and regulatory bodies, IMPACT-AML aims to set new standards in AML treatment and foster open science through data sharing and collaboration.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

In the first 18 months, a major achievement of the IMPACT-AML project has been the finalization and launch of the STREAM platform, which standardizes clinical data collection for R/R AML patients. This platform enables the collection of comprehensive, high-quality patient data, with the first patient already enrolled, marking a significant milestone for research efforts.

Another key accomplishment is the design of the Randomized Pragmatic Clinical Trial (RPCT), aimed at comparing different intensities of rescue therapies for R/R AML. The trial protocol was collaboratively developed by clinical experts, patient representatives, and statisticians to ensure inclusivity and scientific rigor. It has been submitted to Ethics Committees, and the trial is set to begin in December.

The establishment of SOPs for tissue biobanking and MRD analysis was another important milestone, providing a unified framework for collecting and storing biological samples. These SOPs will support translational research and deepen biological insights into AML.

Progress has also been made towards involving underrepresented groups. A study focused on pediatric AML has been designed and initiated, aiming to improve understanding of the disease in children. Discussions have also begun on how best to include neglected cohorts in the research.

The electronic Case Report Form (eCRF) for the RPCT has been developed and finalized, ensuring data quality and compliance with international standards. This tool is vital for accurate data capture and monitoring during the trial.

These achievements lay a strong foundation for future clinical trials and contribute significantly to advancing AML research and improving patient outcomes.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

The development of the STREAM platform is a major breakthrough, enabling standardized data collection for R/R AML patients. STREAM enhances data quality and accessibility, facilitating better analysis and application of insights. This infrastructure will drive accurate, real-world outcomes across diverse patient populations.

Another key achievement is the design of the first RPCT, comparing rescue therapies for R/R AML. The RPCT focuses on inclusivity and real-world application, ensuring results are highly relevant for clinical practice.

The establishment of standardized biobanking and MRD procedures ensures consistent sample collection and analysis across clinical centers, enhancing the reliability of research outcomes and supporting translational studies.

IMPACT-AML has also prioritized inclusivity by focusing on underrepresented groups, such as pediatric patients and neglected cohorts in disadvantaged areas, ensuring comprehensive research benefits all patient groups.

The project has initiated global outreach by collaborating with networks like the African BMT Network and LatBMT Network, as well as engaging with EUAfrica PerMed. These efforts extend AML research beyond Europe, reducing disparities.

To support further uptake, follow-up research and real-world demonstration are needed to validate STREAM and RPCT findings. Partnerships with biotech and pharmaceutical companies are essential for leveraging the platform for new trials and ensuring sustainability. Legal support for intellectual property and data sharing, along with continued investment in training, will be crucial for scaling these innovations.

IMPACT-AML has taken important steps toward improving AML care and research. Ongoing efforts are essential to scale these innovations and benefit AML patients globally.

Final logo of the project